Arikayce (amikacin liposome inhalation suspension) / Insmed - LARVOL DELTA

Trial Conduct, Baseline Characteristics, and Symptom Burden of Patients in the ARISE Study. (PubMed, Pulm Ther) - P3 | "Patients in ARISE were representative of a real-world patient population with MACLD. Comorbid chronic respiratory diseases were common in patients with new or recurrent MACLD, and substantial disease burden at the time physicians initiated MACLD treatment was evidenced by impairment across measures of fatigue and QOL-B domains."

Journal • Asthma • Bronchiectasis • Chronic Obstructive Pulmonary Disease • Fatigue • Immunology • Infectious Disease • Nontuberculous Mycobacterial Disease • Pulmonary Disease • Respiratory Diseases

Healthcare Resource Utilization Associated With Treatment for Refractory Mycobacterium Avium Complex Lung Disease: Comparison of Amikacin Liposome Inhalation Suspension (ALIS) vs Non-ALIS Antibiotic Treatment (ATS 2025) - No abstract available

HEOR • Pulmonary Disease • Respiratory Diseases

Insmed Reports Fourth-Quarter and Full-Year 2024 Financial Results and Provides Business Update (PRNewswire) - "ARIKAYCE global revenue grew 19% in 2024 compared to 2023...The Company continues to anticipate a topline readout for ENCORE in the first quarter of 2026, with the submission of a supplementary new drug application (sNDA) to the FDA for ARIKAYCE in all patients with MAC lung disease in the U.S. expected later in 2026...Regulatory submissions for brensocatib in the EU, UK, and Japan are planned for 2025, with commercial launches anticipated in 2026...The Phase 2b BiRCh study of brensocatib in patients with chronic rhinosinusitis without nasal polyps (CRSsNP) remains on track to report topline results by the end of 2025...The Company plans to initiate a Phase 3 study of TPIP in patients with PH-ILD in the second half of 2025. Enrollment in the Phase 2b study of TPIP in pulmonary arterial hypertension (PAH) completed in December 2024...Insmed continues to anticipate topline data from the study in the middle of 2025."

Commercial • Filing • New P3 trial • P2b data • P3 data: top line • Infectious Disease • Nasal Polyps • Pulmonary Arterial Hypertension • Pulmonary Disease

ENCORE: Study to Evaluate ALIS (Amikacin Liposome Inhalation Suspension) in Participants With Nontuberculous Mycobacterial Lung Infection Caused by Mycobacterium Avium Complex (clinicaltrials.gov) - P3 | N=425 | Active, not recruiting | Sponsor: Insmed Incorporated | Recruiting ➔ Active, not recruiting

Enrollment closed • Nontuberculous mycobacteria • Infectious Disease • Nontuberculous Mycobacterial Disease • Respiratory Diseases

Outcomes and characteristics of patients treated with nebulised amikacin liposome inhalation suspension (Arikayce®): report from a tertiary centre (BTS WM 2024) - "Am J Crit Care Med 2018; 198 (12):1559–1569. View this table: View inline View popup Abstract S44 Table 1 Comparison of responders vs. non-responders"

Clinical • Bronchiectasis • Inflammation • Non‐Cystic Fibrosis Bronchiectasis • Pulmonary Disease • Respiratory Diseases

LONGER USE OF AMIKACIN LIPOSOMAL INHALATION SUSPENSION DURING FIRST 12 MONTHS ASSOCIATED WITH LOWER RISK OF HOSPITALIZATIONS AND EMERGENCY ROOM VISITS: A CLAIMS ANALYSIS (CHEST 2024) - "PURPOSE: Amikacin liposome inhalation suspension (ALIS, Arikayce®) with multidrug regimen (MDR) is the only therapy approved for treatment of Mycobacterium avium complex lung disease (MACLD) in patients who do not achieve culture conversion (CC) after ≥6 consecutive months of MDR. Patients with longer use of ALIS use during the first 12 months following initiation were less likely to experience hospitalization or ER visits at follow-up than patients with shorter use. CLINICAL IMPLICATIONS: The health care resource utilization outcomes observed with longer ALIS use in this study is worth considering when tailoring treatment plans for patients with refractory MACLD. However, more work is needed to understand severity of disease and other confounding factors that may impact ALIS use and outcomes."

Clinical • Asthma • Cardiovascular • Chronic Obstructive Pulmonary Disease • Cough • Diabetes • Dysphonia • Immunology • Infectious Disease • Metabolic Disorders • Pneumonia • Pulmonary Disease • Respiratory Diseases

ENCORE: Study to Evaluate ALIS (Amikacin Liposome Inhalation Suspension) in Participants With Nontuberculous Mycobacterial Lung Infection Caused by Mycobacterium Avium Complex (clinicaltrials.gov) - P3 | N=400 | Recruiting | Sponsor: Insmed Incorporated | N=250 ➔ 400 | Trial completion date: Aug 2023 ➔ Jan 2026 | Trial primary completion date: Aug 2023 ➔ Nov 2025

Enrollment change • Nontuberculous mycobacteria • Trial completion date • Trial primary completion date • Infectious Disease • Nontuberculous Mycobacterial Disease • Respiratory Diseases

Amikacin-Liposome Inhalation Suspension (ALIS) for Refractory MACLD: Simultaneous Analysis of Efficacy and Safety (IDWeek 2024) - No abstract available

Clinical • Infectious Disease • Tuberculosis

ACUTE INTERSTITIAL PNEUMONIA SECONDARY TO AMIKACIN LIPOSOME INHALATION SUSPENSION (CHEST 2024) - "CASE PRESENTATION: A 78-year-old female with chronic mycobacterium avium, previously treated with ethambutol, rifampin, and azithromycin for 1 year and 5 months, was prescribed inhaled Arikayce (amikacin) which led to acute interstitial pneumonia...Amikacin was immediately discontinued, and the patient was started on 60mg dose of prednisone daily – which has gradually been tapered over 5 months to 10mg of prednisone... ALIS has been approved as adjunctive therapy for the treatment of refractory pulmonary MAC infection. The report details a severe adverse respiratory complication. This patient's symptoms occurred rapidly after the initiation of ALIS."

Bronchiectasis • Cough • Dysphonia • Fatigue • Fibrosis • Immunology • Infectious Disease • Interstitial Lung Disease • Pneumonia • Respiratory Diseases

Insmed Reports Second-Quarter 2024 Financial Results and Provides Business Update (PRNewswire) - "Insmed looks forward to presenting additional data from the ASPEN study, including prespecified subpopulation data, at CHEST 2024, taking place October 6-9 in Boston...The Company continues to enroll patients in the Phase 2b BiRCh trial of brensocatib in patients with chronic rhinosinusitis without nasal polyps (CRSsNP) and anticipates providing topline data from the study in the second half of 2025; The Company anticipates activating the first U.S. sites in its Phase 2 study of brensocatib in patients with hidradenitis suppurativa (HS) by the end of 2024....Insmed continues to anticipate that over 80% of total expenditures will be on its mid- to late-stage and commercial programs (ARIKAYCE, brensocatib, and TPIP), and that less than 20% of overall spend will be on its early-stage research programs, reflecting the Company's historical approach to spending."

Commercial • New P2 trial • P2b data • P3 data • Trial status • Bronchiectasis • Hidradenitis Suppurativa • Immunology • Interstitial Lung Disease • Pulmonary Disease • Respiratory Diseases

Qualitative Interviews Exploring Adverse Event Mitigation Strategies in Adults Receiving Amikacin Liposome Inhalation Suspension. (PubMed, Pulm Ther) - "This real-world study identified a diverse set of potential AE mitigation strategies intended to help individual patients prepare for ALIS treatment, prevent the increased emergence of certain AEs, and mitigate the impact of AEs on treatment persistence. Developing a comprehensive accounting of the types of mitigation strategies in use among patients in real-world settings can inform future investigation of the effectiveness of such strategies, and support evidence-based recommendations for treatment management."

Adverse events • Interview • Journal • Fatigue • Nontuberculous Mycobacterial Disease • Pulmonary Disease • Respiratory Diseases

Insmed Announces Pricing of $650 Million Public Offering of Common Stock (PRNewswire) - "Insmed Incorporated...today announced that it priced an underwritten public offering of 12,621,359 shares of its common stock at a price to the public of $51.50 per share...The gross proceeds to Insmed from the offering, before deducting underwriting discounts and commissions and other offering expenses payable by Insmed, are expected to be approximately $650 million....Insmed intends to use the net proceeds from this offering to fund continued research and development of brensocatib as well as pre-commercial and, if approved, commercialization activities related to brensocatib, activities related to the further commercialization and development of ARIKAYCE (amikacin liposome inhalation suspension), further research and development of treprostinil palmitil inhalation powder (TPIP) and any of the Company's other research product candidates, and for other general corporate purposes, including business expansion activities."

Financing • Bronchiectasis • Cystic Fibrosis • Immunology • Oncology • Pulmonary Disease • Respiratory Diseases

Microbiologic Outcomes From a Randomized, Double-blind Trial of Amikacin Liposome Inhalation Suspension in Adults With Newly Diagnosed or Recurrent Mycobacterium Avium Complex Lung Disease: The ARISE Study (ATS 2024) - P3 | " Adults with non-cavitary MACLD who had not received antibiotic therapy for their present infection were randomized 1:1 to receive either ALIS (ALIS arm) [590 mg] or empty liposome control (comparator arm), on a background of azithromycin [250 mg] and ethambutol [15 mg/kg] once daily for 6 months, followed by 1 month off all treatment...No patient developed a MAC isolate with resistance to amikacin and/or clarithromycin... Patients with new or recurrent MACLD who received 6 months of ALIS added to a macrolide-based regimen had numerically greater rates of culture conversion by Months 6 and 7 vs the comparator arm. Time to conversion was faster in the ALIS arm. No new safety signals were identified."

Clinical • Late-breaking abstract • Bronchiectasis • Cough • Dysphonia • Infectious Disease • Nontuberculous Mycobacterial Disease • Pulmonary Disease • Respiratory Diseases

Change in Patient Reported Respiratory Symptoms in a Randomized, Double-blind, Trial of Amikacin Liposome Inhalation Suspension in Adults With Newly Diagnosed or Recurrent Mycobacterium Avium Complex Lung Disease: The ARISE Study (ATS 2024) - P3 | " Adults with non-cavitary MACLD who had not received antibiotic therapy for their present infection were randomized 1:1, receiving ALIS (ALIS arm) [590 mg] or empty liposome control (comparator arm), plus azithromycin [250 mg] and ethambutol [15 mg/kg]... Patients with new or recurrent MACLD who received 6 months of ALIS added to a macrolide-based regimen were able to achieve the estimated meaningful score improvement in respiratory symptoms based on QOL-B RD, with a clear separation between the ALIS and comparator arms. A 15-month confirmatory study (12 months on-treatment, 3 months off-treatment) is ongoing (ENCORE; NCT04677569)."

Clinical • Bronchiectasis • Infectious Disease • Nontuberculous Mycobacterial Disease • Pulmonary Disease • Respiratory Diseases

Insmed Presents Positive Patient-Reported Outcomes and Microbiologic Data from Phase 3 ARISE Study Of ARIKAYCE (Amikacin Liposome Inhalation Suspension) in Patients with NTM Lung Disease Caused by MAC at American Thoracic Society 2024 International Conference Plenary Session (Insmed Press Release) - P3 | N=99 | ARISE (NCT04677543) | Sponsor: Insmed Incorporated | "Data from ARISE evaluating patients with newly diagnosed or recurrent nontuberculous mycobacterial (NTM) lung infection caused by Mycobacterium avium complex (MAC) who had not received antibiotics for their current infection were presented in an oral session and during the ATS Breaking News session 'Clinical Trial Results in Pulmonary Medicine.'...In ARISE, ARIKAYCE-treated patients performed better than those in the comparator arm (a macrolide-based multi-drug regimen) as measured by the QOL-B instrument, with 43.8% of patients achieving an improvement in QOL-B respiratory score above or equal to the estimated meaningful within-subject score difference of 14.8, compared with 33.3% of patients in the comparator arm....New data presented showed that patients in the ARIKAYCE treatment arm experienced continued improvement in QOL-B scores to Month 7..."

P3 data • Patient reported outcomes • Infectious Disease • Nontuberculous Mycobacterial Disease

Psychometric Validation of the PROMIS Fatigue-Short Form 7A in Adults with Newly Diagnosed or Recurrent Mycobacterium avium Complex (MAC) Lung Disease: The ARISE and ENCORE Studies (ISPOR 2024) - P3 | " Data analyzed were from ARISE (NCT04677543; baseline and longitudinal data) and ENCORE (NCT04677569; baseline data) double-blind, randomized, active-control trials assessing impact of once-daily amikacin liposome inhalation suspension (ALIS) treatment on fatigue symptoms in MAC-LD... The findings demonstrate the PROMIS F-SF 7a has adequate reliability, validity, and responsiveness for assessing fatigue symptoms in adults with newly diagnosed or recurrent MAC-LD."

Clinical • Fatigue • Pulmonary Disease • Respiratory Diseases

Psychometric Validation of the QOL-B Respiratory Domain in Adults with Newly Diagnosed or Recurrent Mycobacterium avium Complex (MAC) Lung Disease: The ARISE and ENCORE Studies (ISPOR 2024) - P3 | " Data analyzed were from ARISE (NCT04677543; baseline and longitudinal data) and ENCORE (NCT04677569; baseline data) double-blind, randomized, active-control trials assessing the impact of once-daily amikacin liposome inhalation suspension (ALIS) treatment on respiratory symptoms in MAC-LD... The findings demonstrate the QOL-B-RD has adequate reliability, validity, and responsiveness for assessing respiratory symptoms in adults with newly diagnosed or recurrent MAC-LD."

Clinical • Bronchiectasis • Fatigue • Infectious Disease • Pulmonary Disease • Respiratory Diseases

Insmed to Present New Data from Across its Respiratory Portfolio at the American Thoracic Society 2024 International Conference (PRNewswire) - "Insmed Incorporated...today announced that it will present nine abstracts from across its respiratory portfolio at the American Thoracic Society (ATS) 2024 International Conference, taking place May 17-22, 2024, in San Diego. 'Further, we look forward to sharing data from across our respiratory portfolio, including brensocatib in bronchiectasis and data on the burden of illness of pulmonary hypertension associated with interstitial lung disease, as we advance our efforts to bring forward novel, urgently needed therapies.'"

P2 data • Bronchiectasis • Non‐Cystic Fibrosis Bronchiectasis

A qualitative interview study to explore the use of adverse event mitigation strategies among adults receiving amikacin liposome inhalation suspension (ALIS) in real world settings (IWLH 2024) - "Mitigation strategies intended to prepare patients for ALIS treatment, prevent the increased emergence of certain AEs, and mitigate impact of AEs on treatment persistence may have clinical relevance for treatment of MAC-LD with ALIS. This study collected real-world data from patients to identify the diverse set of AE mitigation strategies used by patients and identified potential opportunities clinicians can avail of and adopt in improving adherence to treatment with ALIS. These qualitative data can inform future studies to further quantify the effectiveness of different AE mitigation strategies in real-world settings."

Adverse events • Clinical • Interview • Real-world • Real-world evidence • Fatigue • Pulmonary Disease • Respiratory Diseases

Single Dose Escalation Study of TR02 (Sustained Lipid Inhalation Technology [SLIT™] Amikacin) in Participants With Cystic Fibrosis (CF) Having Chronic Infections of Pseudomonas Aeruginosa (clinicaltrials.gov) - P1 | N=18 | Completed | Sponsor: Insmed Incorporated

New P1 trial • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Infectious Disease • Pulmonary Disease • Respiratory Diseases

Amikacin liposome inhalation suspension for Mycobacterium avium complex pulmonary disease: A subgroup analysis of Japanese patients in the randomized, phase 3, CONVERT study. (PubMed, Respir Investig) - P3 | "The efficacy observed in the Japanese subpopulation was largely consistent with that in the overall CONVERT study population, with more patients achieving culture conversion with ALIS + GBT versus GBT alone. Safety profiles were similar between the overall population and the Japanese subpopulation."

Journal • P3 data • Bronchiectasis • Nontuberculous Mycobacterial Disease • Pulmonary Disease • Respiratory Diseases

REAL-WORLD EXPERIENCE WITH NEBULISED AMIKACIN LIPOSOME INHALATION SUSPENSION (ARIKAYCE®): REPORT FROM A TERTIARY CENTRE (BTS WM 2023) - "Discussion Treatment of refractory patients with MAC is challenging. To date 1/3 patients on Arikayce for 6 mts culture converted, reflecting the CONVERT study.1 Side effects were commonly reported, but most patients were able to continue the medication."

Clinical • Real-world • Real-world evidence • Bronchiectasis • Chronic Obstructive Pulmonary Disease • Cough • Dysphonia • Immunology • Pulmonary Disease • Respiratory Diseases

A QUALITATIVE INTERVIEW STUDY TO EXPLORE THE USE OF ADVERSE EVENT MITIGATION STRATEGIES AMONG ADULTS RECEIVING AMIKACIN LIPOSOME INHALATION SUSPENSION (ALIS) IN REAL WORLD SETTINGS (BTS WM 2023) - "Real-world data identified the diverse set of AE mitigation strategies used by patients and also opportunities clinicians can avail of and adopt in improving adherence to ALIS treatment. These qualitative data can inform future studies to further quantify the effectiveness of AE mitigation strategies in realworld settings."

Adverse events • Clinical • Interview • Real-world • Real-world evidence • Fatigue • Pulmonary Disease • Respiratory Diseases

INSMED ANNOUNCES POSITIVE TOPLINE RESULTS FROM PHASE 3 ARISE STUDY OF ARIKAYCE (AMIKACIN LIPOSOME INHALATION SUSPENSION) IN PATIENTS WITH NTM LUNG DISEASE CAUSED BY MAC (Insmed Press Release) - P3 | N=99 | ARISE (NCT04677543) | Sponsor: Insmed Incorporated | "Insmed Incorporated...announced positive topline results from its Phase 3 ARISE study of ARIKAYCE in patients with newly diagnosed or recurrent nontuberculous mycobacterial (NTM) lung infection caused by Mycobacterium avium complex (MAC) who had not started antibiotics...The study met its primary objective of demonstrating that the Quality of Life – Bronchiectasis (QOL-B) respiratory domain works effectively as a patient-reported outcome (PRO) instrument in patients with MAC lung disease. Based on these results, Insmed plans to propose to the U.S. Food and Drug Administration (FDA) that the QOL-B respiratory domain PRO be the primary endpoint for the ENCORE study without any modifications...ARIKAYCE-treated patients performed better than those in the comparator arm as measured by the QOL-B instrument, with 43.8% of patients achieving an improvement in QOL-B respiratory score..."

P3 data: top line • Patient reported outcomes • Nontuberculous Mycobacterial Disease

A Study to Investigate Lung Deposition of Inhaled Amikacin-loaded Liposomes in Healthy Male Participants (clinicaltrials.gov) - P1 | N=6 | Completed | Sponsor: Insmed Incorporated

New P1 trial